FDA to Host Patient-Focused Drug Development Meeting for FA Community

Patients with Friedreich’s ataxia (FA) and caregivers may benefit from participating in the upcoming Patient-Focused Drug Development meeting by the U.S. Food and Drug Administration (FDA).

The meeting will be held at the College Park Marriott and Conference Center, Hyattsville, Maryland, on June 2 (8 a.m.-12:30 p.m.). After the meeting, there will be a research update for anyone who is interested from 1:30-3 p.m..

The Patient-Focused Drug Development meetings allow the FDA to hear directly from the community what are patients’ needs and what is their experience of living with specific diseases. These meetings also provide an opportunity to talk about the impact of potential treatments and the risks/benefits of new medications.

“The Patient Focused Drug Development Meeting is your opportunity to tell the FDA and drug developers what matters to you about living with FA, including symptoms that are most troublesome, and your vision for potential meaningful therapies,” says the Friedreich’s Ataxia Research Alliance (FARA).

“The FDA will use that information as it evaluates the potential benefits of a therapy to the FA patient population, relative to any potential risks. This is your opportunity to describe your view of a successful therapy, and how it would improve your quality of life. The report will summarize the input from patients across the country, so we encourage everyone to get involved, whether through in-person attendance at the meeting, on-line attendance and voting or through participation in surveys we send out to gather data prior to the meeting.”

FARA encourages all interested patients, family and caregivers to participate in the meeting. For those who cannot attend in person, there will be an online streaming of the meeting, which participants can use to vote on specific panel questions and provide other information that will be useful to build a report on the outcome of the meeting to the FDA.

According to FARA, after the meeting, the in-person and online answers to the panel questions, as well as data collected before the meeting, will be included in a “Voice of the Patient” report, which will be submitted to the FDA.

FA is an incurable neurodegenerative disease that derives from a mutation in the frataxin protein (caused by repeats of a DNA sequence, known as GAA repeat expansion), which impairs its normal expression. These expansions cause a substantial reduction in frataxin protein levels and several consequences at cellular level.